Keystone Symposium „From Rare to Care: Discovery, Modeling and Translation of Rare Diseases”
- 11 November 2018 - 14 November 2018
- Vienna BioCenter, IMP Lecture Hall, Vienna, Austria
Work over the past 30 years has resulted in the identification of genes for approximately 50 percent of the estimated 7,000 rare genetic diseases; it is predicted that most of the remaining disease genes will be identified in the next 10 years. Approximately 500 medicinal products are currently on the market for rare diseases. The accelerating pace of rare disease gene identification means, in effect, an almost commensurate increase in molecularly defined, readily diagnosable, but nonetheless poorly understood and untreatable, diseases. This meeting will examine the current and future bottlenecks to gene discovery, disease modeling, and therapeutic approaches and suggest strategies to enable progress in this regard. Ultimately, successful deployment of precision medicine for rare diseases will inform such approaches more broadly.
The meeting will begin on Sunday, November 11 with registration from 16:00 to 20:00 and a welcome mixer from 18:00 to 20:00. Conference events conclude on Wednesday, November 14 with a closing plenary session and keynote address from 16:00 to 18:30, followed by a social hour. We recommend return travel on Thursday, November 15 in order to fully experience the meeting.
Scientific organisers: Josef Penninger, Kym Boycott